the glocalized network in theragenomic and personalized medicine and the genetics of individual patients safety of drugs
March 15, 2024 – On the occasion of International Rare Disease Day on February 28, the Swiss Foundation for People with Rare Diseases drew attention to the current challenges and needs of those affected. In Europe, a disease is considered …
Rare diseases: No diagnosis or the most expensive drug in the world? Read more »
August 13, 2019 – Onasemnogene abeparvovec-xioi (Zolgensma) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 …
Onasemnogene abeparvovec-xioi (Zolgensma): Approval strategy with manipulated data? Read more »
May 29, 2019 – Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. The severity of symptoms and age of onset varies by the type. Some types are apparent at …
Gene therapy: Zolgensma to treat spinal muscular atrophy Read more »